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Treatment-related benefit and satisfaction in patients with Fabry disease in France: insight into patients’ expectations and preferences from the prospective, non-interventional SATIS-Fab study

Abstract Background Fabry disease (FD) is a progressive X-linked lysosomal disorder caused by GLA variants resulting in deficient α-galactosidase A enzyme activity, glycolipid accumulation, and multisystemic dysfunction. Approved treatments include intravenous enzyme replacement therapy (ERT) or the...

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Principais autores: Olivier Lidove, Agathe Masseau, Grégory Pugnet, Didier Lacombe, Bertrand Dussol, Soumeya Bekri, Albert Hagège, Caroline Martinez, Yann Fardini, Alain Fouilhoux, Esther Noël
Format: Artigo
Sprog:Inglês
Udgivet: BMC 2026-04-01
Serier:Orphanet Journal of Rare Diseases
Fag:
Fabry disease
Migalastat
Enzyme replacement therapy
Treatment expectations
Treatment satisfaction
Patient Benefit Index
Online adgang:https://doi.org/10.1186/s13023-026-04285-7
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